Judy Weisenberg, MD

Professor of Neurology, Division of Pediatric Neurology, Epilepsy Section

Co-Director Washington University Rett Spectrum Clinic

Washington University School of Medicine

Talk Title: An Aerial View of Genetic Targeted Therapies in Neurodevelopmental Disabilities in 2026: Progress, Pitfalls and Conundra

Abstract: In the past 2 decades, there have been dramatic advances in our understanding of the genetics of many forms of neurodevelopmental disability as well as our ability to diagnose and potential treatment strategies.  This talk will focus on Dravet Syndrome and Rett Syndrome but also touch on other similar conditions and the current state of the clinical trials as well as lessons learned and ongoing challenges.  

Host: Dr. Joe Dougherty

Host comments: Dr. Weisenberg is an award winning pediatric neurologist and epileptologist with a deep interest in genetic forms of neurodevelopmental disorders. She is Professor of Neurology here at Washington University, and on top doctors in pediatric epilepsy across the region.  Over the years, she has made substantial contributions to discovering genetic causes, characterizing the consequences of specific gene mutations in humans, and developing gene-informed treatments.  Critically, she has co-led the Rett Spectrum clinic here on campus since 2013, and is deeply involved in clinical trials using gene therapy for Rett Syndrome – a previously untreatable severe and regressive neurodevelopmental disorder.  She will share with us her overview of the landscape of cutting edge trials, emerging treatments, pitfalls and conundra when attempting gene therapy for human disorders of neurodevelopment.